Alexion Pharmaceuticals, Inc. (ALXN) announced that the U.S. Food and Drug Administration (FDA) has approved Kanuma (sebelipase alfa) for the treatment of patients of all ages with a diagnosis of lysosomal acid lipase deficiency or LAL-D.
Kanuma, an enzyme replacement therapy (ERT), is the first therapy approved in the U.S. for the treatment of patients with LAL-D, a genetic and progressive ultra-rare metabolic disease in which patients suffer multi-organ damage and premature death.
Alexion said it is preparing to serve patients in the U.S. with Kanuma and expects that Kanuma will become available commercially during the first week of January 2016. The Company's expanded access program will remain open to enable patients with LAL-D in the U.S. to access Kanuma until commercial product is available.
LAL-D is a genetic, chronic, and progressive metabolic disease associated with significant morbidity and premature mortality. It is an ultra-rare disease, which is defined as a disease that affects fewer than 20 patients per one million of the general population.
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